X4 Pharmaceuticals Announces Key Enrollment Milestone Achievements in Ongoing Mavorixafor Clinical Trials and Reports Second Quarter Financial Results
Twenty-three patients now enrolled in ongoing pivotal Phase 3 trial in WHIM syndrome, surpassing minimum needed for primary endpoint analyses; enrollment to complete in 3Q21 with top-line data expected in 4Q22
Minimum number of patients now enrolled to determine optimal dosing of mavorixafor in Waldenström’s macroglobulinemia Phase 1b trial; continue to expect high-dose safety and efficacy data, as well as clinical response outcomes, in 4Q21
Initial data from Phase 1b trial in Severe Congenital Neutropenia (SCN) expected in 4Q21, as company explores expanded use of mavorixafor across broader chronic neutropenia populations
Conference call today at
“We are very encouraged by the strong interest from both the physicians and patients participating in our mavorixafor clinical programs,” said
Mavorixafor Clinical Trial Updates
- Phase 3 Trial in WHIM Syndrome (4WHIM):
- The company today announced that it has surpassed the 18-patient minimum enrollment needed for primary endpoint analyses, determination of clinical benefit, and
U.S. regulatory filing (if supported by the Phase 3 data), having enrolled 23 patients to date in its ongoing Phase 3 trial in WHIM syndrome. Enrollment will be completed in the third quarter of 2021, allowing the remaining identified patients to complete screening and potential enrollment. Top-line data are expected to be announced in the fourth quarter of 2022. - The 4WHIM Phase 3 trial is a global, randomized, double-blind, placebo-controlled, multicenter study designed to evaluate the safety and efficacy of mavorixafor in 18-28 genetically confirmed WHIM patients over the course of a 52-week study with open-label extension. The primary endpoint for the trial will compare the level of circulating neutrophils relative to a clinically meaningful threshold in response to treatment with mavorixafor versus placebo over 24-hour periods. Secondary endpoints will assess infection rates, wart burden, markers of immune system function, and quality of life, among others.
- The company is planning to announce new data from the open-label extension of its ongoing Phase 2 clinical trial, as well as an update on patient prevalence, and new data from research into the genetics of WHIM that will detail new insights into genotype/phenotype correlations and the identification of a new WHIM variant.
- The company today announced that it has surpassed the 18-patient minimum enrollment needed for primary endpoint analyses, determination of clinical benefit, and
- Phase 1b Trial in Waldenström’s Macroglobulinemia (WM):
- The company also announced today that it has surpassed enrollment of the minimum 12 patients (Cohorts A and B) required to determine optimal dosing of mavorixafor in combination with ibrutinib in the ongoing Phase 1b clinical trial. The company is continuing enrollment in the optional Cohort C (up to an additional 6 patients).
- This ongoing Phase 1b, open-label, multicenter, single-arm study examines intra-patient dose escalation, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of mavorixafor (200 mg, 400 mg, and 600 mg) in combination with ibrutinib (420 mg), both delivered orally once daily, in patients with Waldenström’s macroglobulinemia and confirmed MYD88 and CXCR4 mutations. Patients are followed for adverse events and change from baseline in IgM and hemoglobin, PK, and PD (including peripheral white blood cell counts), in addition to clinical response.
- The company remains on track to announce additional dosing, efficacy, safety, and clinical response data from the ongoing trial in the fourth quarter of 2021, including patient data at the highest planned mavorixafor dose of 600 mg.
- Phase 1b Trial in Severe Congenital Neutropenia (SCN):
- Enrollment continues in this clinical trial, with initial data anticipated in the fourth quarter of 2021. The company expects that the initial data from this trial, in combination with additional data emerging from prior and ongoing studies that show chronic, sustained white blood cell increases across a number of patient groups treated with mavorixafor, will support the company’s exploration of opportunities for mavorixafor use across larger chronic neutropenic populations and more broadly in cellular immunodeficiencies.
Second Quarter Highlights and Upcoming Events
- EHA 2021: In June, the company announced the presentation of positive data from its ongoing Phase 1b clinical trial of mavorixafor in combination with ibrutinib in Waldenström’s macroglobulinemia. Data showed robust decreases in serum IgM at low- and mid-doses of mavorixafor, suggesting best-in-class potential for this combination treatment; meaningful increases in hemoglobin levels suggested reduction in cancer burden in the bone marrow; and at 6 months, patients achieved median IgM level reductions of 60%-75%, with one patient achieving normal IgM; two of four patients (50%) had >50% reduction in serum IgM from baseline. The poster is available here; slides from the company’s associated analyst event are available here.
- X4 management will be participating in the following upcoming investor conferences:
Canaccord Genuity Growth Conference – taking place virtuallyAugust 10-12, 2021 - Citi Annual BioPharma Conference– taking place virtually
September 8-10, 2021 - Oppenheimer Fall Healthcare Life Science & Med Tech Summit – taking place virtually
September 20-23, 2021 Cantor Fitzgerald Healthcare Conference – taking place virtuallySeptember 27-30, 2021
Second Quarter 2021 Financial Results
- Cash, Cash Equivalents & Restricted Cash: X4 had
$96.5 million in cash, cash equivalents, and restricted cash as ofJune 30, 2021 . The company expects that its cash and cash equivalents will fund company operations into the fourth quarter of 2022. - Research and Development Expenses were
$13.2 million for the second quarter endedJune 30, 2021 , as compared to$9.3 million for the comparable period in 2020. R&D expenses include$0.8 million and$0.5 million of certain non-cash expenses for the quarters endedJune 30, 2021 and 2020, respectively. - General and Administrative Expenses were
$5.8 million for the second quarter endedJune 30, 2021 , as compared to$5.3 million for the comparable period in 2020. G&A expenses include$1.0 million and$0.7 million of certain non-cash expenses for the quarters endedJune 30, 2021 and 2020, respectively. - Net Loss: X4 reported a net loss of
$19.6 million for the quarter endedJune 30, 2021 , as compared to a net loss of$15.1 million for the comparable period in 2020. Net losses include$1.8 million and$1.2 million of certain non-cash expenses for the quarters endedJune 30, 2021 and 2020, respectively.
Conference Call and Webcast
X4 will host a conference call and webcast today at
About
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by the words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target,” or other similar terms or expressions that concern X4's expectations, strategy, plans, or intentions. Forward-looking statements include, without limitation, statements regarding the clinical development and therapeutic potential of mavorixafor and X4’s other product candidates or programs; X4’s possible exploration of additional opportunities for mavorixafor; the anticipated achievement of upcoming clinical milestones; the expected availability, content, and timing of clinical trial data; anticipated regulatory filings; clinical trial design, and the company’s cash runway. Any forward-looking statements in this press release are based on management's current expectations and beliefs. Actual events or results may differ materially from those expressed or implied by any forward-looking statements contained herein, including, without limitation, uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development; the risk that trials and studies may be delayed, including, but not limited to, as a result of the effects of the ongoing COVID-19 pandemic or delayed patient enrollment, and may not have satisfactory outcomes; the risk that the outcomes of preclinical studies or earlier clinical trials will not be predictive of later clinical trial results; the risk that initial or interim results from a clinical trial may not be predictive of the final results of the trial or the results of future trials; the potential adverse effects arising from the testing or use of mavorixafor or other product candidates; risks related to X4’s ability to raise additional capital and other risks and uncertainties, including those described in the section entitled “Risk Factors” in X4’s Quarterly Report on Form 10-Q filed with the
(Tables Follow)
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
(in thousands, except per share amounts)
(unaudited)
Three Months Ended | Six Months Ended | ||||||||||||||
2021 | 2020 | 2021 | 2020 | ||||||||||||
License revenue | $ | — | $ | — | $ | — | $ | 3,000 | |||||||
Operating expenses: | |||||||||||||||
Research and development | 13,193 | 9,342 | 25,297 | 18,253 | |||||||||||
General and administrative | 5,804 | 5,316 | 11,636 | 9,986 | |||||||||||
Total operating expenses | 18,997 | 14,658 | 36,933 | 28,239 | |||||||||||
Loss from operations | (18,997 | ) | (14,658 | ) | (36,933 | ) | (25,239 | ) | |||||||
Other expense, net | (635 | ) | (486 | ) | (1,369 | ) | (895 | ) | |||||||
Loss before provision for income taxes | (19,632 | ) | (15,144 | ) | (38,302 | ) | (26,134 | ) | |||||||
Provision for income taxes | 6 | — | 12 | 148 | |||||||||||
Net loss | (19,638 | ) | (15,144 | ) | (38,314 | ) | (26,282 | ) | |||||||
Deemed dividend due to Class B warrant price reset | — | — | (8,239 | ) | — | ||||||||||
Net loss attributable to common stockholders | $ | (19,638 | ) | $ | (15,144 | ) | $ | (46,553 | ) | $ | (26,282 | ) | |||
Net loss per share attributable to common stockholders- basic and diluted | $ | (0.74 | ) | $ | (0.76 | ) | $ | (1.97 | ) | $ | (1.31 | ) | |||
Weighted average common shares outstanding-basic and diluted | 26,527 | 20,032 | 23,655 | 20,016 |
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS
(in thousands)
(unaudited)
Six months ended |
|||||||
2021 | 2020 | ||||||
Net loss | $ | (38,314 | ) | $ | (26,282 | ) | |
Adjustments to reconcile net loss to net cash used in operating activities | 4,345 | 2,591 | |||||
Changes in operating assets and liabilities | (3,672 | ) | (3,295 | ) | |||
Net cash used in operating activities | (37,641 | ) | (26,986 | ) | |||
Net cash used in provided by investing activities | (582 | ) | (564 | ) | |||
Net cash provided by financing activities | 54,117 | 5,049 | |||||
Impact of foreign exchange on cash, cash equivalents and restricted cash | (103 | ) | 60 | ||||
Net increase (decrease) in cash, cash equivalents and restricted cash | 15,791 | (22,441 | ) | ||||
Cash, cash equivalents and restricted cash at beginning of period | 80,702 | 128,086 | |||||
Cash, cash equivalents and restricted cash at end of period | $ | 96,493 | $ | 105,645 |
CONSOLIDATED BALANCE SHEETS
(in thousands)
(unaudited)
Current assets: | |||||||
Cash and cash equivalents | $ | 95,161 | $ | 78,708 | |||
Research and development incentive receivable | 1,053 | 917 | |||||
Prepaid expenses and other current assets | 5,157 | 3,682 | |||||
Total current assets | 101,371 | 83,307 | |||||
Property and equipment, net | 1,745 | 1,237 | |||||
27,109 | 27,109 | ||||||
Right-of-use assets | 9,430 | 7,960 | |||||
Other assets | 2,004 | 3,258 | |||||
Total assets | $ | 141,659 | $ | 122,871 | |||
Current liabilities: | |||||||
Accounts payable | $ | 2,528 | $ | 3,144 | |||
Accrued expenses | 9,607 | 8,018 | |||||
Current portion of lease liability | 985 | 786 | |||||
Total current liabilities | 13,120 | 11,948 | |||||
Long-term debt, including accretion, net of discount | 33,542 | 33,178 | |||||
Lease liabilities | 5,350 | 4,484 | |||||
Other liabilities | 434 | 462 | |||||
Total liabilities | 52,446 | 50,072 | |||||
Redeemable common shares | 1,875 | — | |||||
Total stockholders' equity | 87,338 | 72,799 | |||||
Total liabilities, redeemable common shares and stockholders' equity | $ | 141,659 | $ | 122,871 |
Contacts:
Managing Director,
daniel@lifesciadvisors.com; (617) 430-7576
Senior Account Executive,
mroucomolina@lifescicomms.com
Source: X4 Pharmaceuticals